Eltrombopag for the treatment of poor graft function following allogeneic stem cell transplant: a retrospective multicenter study

Sabrina Giammarco, Simona Sica, Patrizia Chiusolo, Luca Laurenti, Federica Sora', Massimo Martino, Michelangelo Martino, Elisabetta Metafuni, Alessandro Busca, Antonio Risitano, Carlos Vallejo, Andrea Bacigalupo

Risultato della ricerca: Contributo in rivistaArticolo in rivista


This retrospective study assessed the effectiveness of eltrombopag (EPAG), a thrombopoietin receptor agonist, in the treatment of poor graft function (PGF) following an allogeneic haemopoietic stem cell transplantation (HSCT). Complete response was defined as normalization of blood counts, whereas partial response was defined as transfusion independence. A total of 48 patients with full donor chimerism after HSCT, received EPAG for a median of 120 days (range 10–591). Patients with uni- bi- or tri-lineage cytopenia started treatment at a median of 95 days (range 17–877) after HSCT. The overall response rate was 75%: 24 patients had a complete response and 12 had a partial response. Positive predictors of response were an HLA-matched donor, a CD34+ dose at transplant > 4 × 106/kg, and starting EPAG treatment at least 90 days after HSCT. Patients with more than one positive predictor had a response rate of 92% for the overall patient cohort and 94% for patients with tri-lineage cytopenia. One-year survival was 89% for complete responders, 60% for partial responders and 20% for non-responders (p = 0.0004). EPAG improves peripheral blood counts in patients with poor graft function following HSCT. Response to EPAG can be predicted and has a significant impact on survival.
Lingua originaleEnglish
pagine (da-a)228-234
Numero di pagine7
RivistaInternational Journal of Hematology
Stato di pubblicazionePubblicato - 2021


  • Adult
  • Aged
  • Benzoates
  • Cytopenia
  • Female
  • Haemopoietic stem cell transplantation eltrombopag
  • Hematologic Diseases
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Hydrazines
  • Italy
  • Male
  • Middle Aged
  • Poor graft function
  • Primary Graft Dysfunction
  • Pyrazoles
  • Retrospective Studies
  • Thrombopoietin receptor agonist
  • Transplantation, Homologous
  • Treatment Outcome
  • Young Adult


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